A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis.
A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis.
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DOI：
10.1007/s40120-020-00208-1
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发表时间：
2020-12
影响因子：
3.7
通讯作者：
Viswanath O
中科院分区：
文献类型：
--
作者：
Urits I;Swanson D;Swett MC;Patel A;Berardino K;Amgalan A;Berger AA;Kassem H;Kaye AD;Viswanath O
遗传性变体甲状腺素运载蛋白淀粉样变性（ATTRv）是一种罕见的遗传缺陷，影响全世界约5000- 10，000人，导致继发于突变甲状腺素运载蛋白（TTR）蛋白原纤维错误折叠的淀粉样变性。TTR突变可导致许多器官细胞外区域的蛋白质沉积，但心脏和轴突细胞中的那些沉积是这种临床综合征的主要原因。治疗选择有限，但正在开发新药。Patisiran是一种新型药物，是一种针对TTR的脂质体siRNA，特异性靶向这种蛋白质，减少TTR在组织中的积累，随后改善神经病变和心脏功能。Patisiran很可能成为开发用于靶向治疗的进一步智能药物解决方案的原型。在这篇综述中，我们总结了目前可用的证据与ATTRv与帕提西兰治疗多发性神经病的人。我们回顾了其有效性，安全性和使用适应症的证据，引用了关于这些主题的新颖和开创性的论文。
Hereditary variant transthyretin amyloidosis (ATTRv) is a rare genetic defect that affects about 5000–10,000 people worldwide, causing amyloidosis secondary to misfolding of mutant transthyretin (TTR) protein fibrils. TTR mutations can cause protein deposits in many extracellular regions of organs, but those deposits in cardiac and axonal cells are the primary cause of this clinical syndrome. Treatment options are limited, but new drugs are being developed. Patisiran, a novel drug, is a liposomal siRNA against TTR that specifically targets this protein, reducing the accumulation of TTR in tissues, with subsequent improvement in both neuropathy and cardiac function. Patisiran is likely to serve as a prototype for the development of further intelligent drug solutions for use in targeted therapy. In this review we summarize the evidence currently available on the treatment of polyneuropathy in people with ATTRv with patisiran. We review the evidence on its efficacy, safety, and indications of use, citing novel and seminal papers on these subjects.
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